A Study of AAV-hAADC-2 in Subjects With Parkinson's Disease
Safety Study in subjects with Parkinson's Disease
|Study Design:||Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase1 Open Label Safety Study of Intrastriatal Infusion of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase (AAV-hAADC-2) in Subjects With Parkinson's Disease [AAV-hAADC-2-003]|
- The safety and tolerability of intrastriatal administration of AAV-hAADC-2 as measured by Adverse Events in subjects with mid- to late-stage Parkinson's Disease. [ Time Frame: Time of treatment through Month 60. ] [ Designated as safety issue: Yes ]
- The effect of AAV-hAADC-2 on clinical status as recorded in subject diaries, by clinical assessment, and daily levodopa requirement. [ Time Frame: Time of treatment through Month 60. ] [ Designated as safety issue: No ]
- The relationship between the dose of AAV-hAADC-2 vector infused and the resulting level of striatal AADC expression by FMT-PET imaging. [ Time Frame: Time of treatment through Month 60 ] [ Designated as safety issue: No ]
|Study Start Date:||November 2004|
|Study Completion Date:||March 2013|
|Primary Completion Date:||March 2013 (Final data collection date for primary outcome measure)|
|Experimental: AAV-hAADC-2 (9x10^10 vector genomes)||
9 x 10^10 vector genomes (vg) of AAV-hAADC-2 in a single dose of 200 µL bilaterally infused over 4 striatal targets
|Experimental: AAV-hAADC-2 (3x10^11 vector genomes)||
3 x 10^11 vector genomes (vg) of AAV-hAADC-2 in a single dose of 200 µL bilaterally infused over 4 striatal targets
In a patient with Parkinson's disease, the part of the brain called the substantia nigra progressively loses the ability to send dopamine signals to the striatum.
To compensate for the loss of striatal dopamine, most patients take L-dopa, an approved drug which is converted to dopamine by an essential enzyme - AADC.
With time, however, the brain loses its remaining ability to convert L-dopa to dopamine and thus the drug becomes progressively less effective.
In the therapy being studied, the gene coding for the enzyme that converts L-dopa to dopamine (AADC) is inserted into a common, non-pathogenic virus (AAV) to which > 90% of humans have been exposed.
The AAV will help to transport the AADC into the brain cells.
AAV-hAADC-2, the investigational drug being studied, is injected into the striatum during a surgical procedure.
Patients who undergo the procedure would continue to take L-dopa; AAV-hAADC-2 is intended to provide, directly to the brain, the missing enzyme needed to convert L-dopa to dopamine.
|United States, California|
|University of California, San Francisco|
|San Francisco, California, United States|
|Study Director:||Medical Monitor||Genzyme, a Sanofi Company|